Deferasirox in Treating Patients With Very Low, Low, or Intermediate-Risk Red Blood Cell Transfusion Dependent Anemia or Myelodysplastic Syndrome

NCT02943668 | Terminated Phase 2 Results DMC FDA Drug

• 3 other identifiers: 9422NCI-2016-01457P30CA015704
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 1

Brief Summary

This phase II trial studies how well deferasirox works in treating patients with very low, low, or intermediate-risk anemia or myelodysplastic syndrome that depends on red blood cell transfusions. Deferasirox may treat too much iron in the blood caused by blood transfusions.

Conditions

Anemia; Myelodysplastic Syndrome

Outcome Measures

Primary Outcomes (1)

• Proportion of Patients That Achieve Erythroid Hematologic Improvement.

  As defined by the modified International Working Group (IWG) response criteria: Erythroid response (pretreatment, \<11 g/dL): 1. Hgb increase by ≥ 1.5 g/dL 2. Relevant reduction of units of RBC transfusions by an absolute number of at least 4 RBC transfusions/8 wk compared with the pretreatment transfusion number in the previous 8 wk. Only RBC transfusions given for a Hgb of ≤ 0.9 g/dL pretreatment will count in the RBC transfusion response evaluation. Platelet response (pretreatment, \< 100 x 10\^9/L) 1. Absolute increase of ≥ 30 x 10\^9/L for patients starting with \> 20 x 10\^9/L platelets 2. Increase from \< 20 x 10\^9/L to \> 20 x 10\^9/L and by at least 100% Neutrophil response (pretreatment, \< 1.0 x 10\^9/L) 1\) At least 100% increase and an absolute increase \> 0.5 x 10\^9/L

  At 6 months

Secondary Outcomes (3)

• Change in Red Blood Cell (RBC) Transfusion Requirements

  Baseline up to 12 months

• Change in Serum Ferritin Levels

  Baseline up to 12 months

• Proportion of Patients Who Achieve Granulocyte or Platelet Hematologic Improvement

  At 6 months

Study Arms (1)

Treatment (deferasirox)

EXPERIMENTAL

Patients receive deferasirox PO QD. Treatment continues for up to 52 weeks in the absence of disease progression or unacceptable toxicity.

Drug: Deferasirox; Other: Laboratory Biomarker Analysis

Interventions

Deferasirox DRUG

Given PO

Also known as: Exjade

Treatment (deferasirox)

Laboratory Biomarker Analysis OTHER

Correlative studies

Treatment (deferasirox)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Capable of giving written informed consent prior to any study-specific procedures
• Diagnosis of MDS as defined by the World Health Organization (WHO) diagnostic criteria
• Have very low, low or intermediate-risk disease by the Revised International Prognostic Scoring System (IPSS-R)
• Baseline serum ferritin level \>= 100 ng/mL
• Have an Eastern Cooperative Oncology Group (ECOG) performance status of 0-2
• Anemia defined as: hemoglobin =\< 10.0 g/dL
• Bilirubin =\< 1.5 times upper limit of normal (ULN)
• Alanine aminotransferase (ALT), and aspartate aminotransferase (AST) =\< 3.5 times ULN
• Serum creatinine =\< 1.5 x ULN
• Estimated glomerular filtration rate (GFR) \> 40 mL/min
• Males and females with reproductive potential must agree to use medically approved contraceptive precautions during the study and for 3 months following the last dose of deferasirox
• Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using effective methods of contraception during dosing of study treatment; effective contraception methods include:
• Placement of an intrauterine device (IUD) or intrauterine system (IUS)
• Barrier methods of contraception: condom or occlusive cap (diaphragm or cervical/vault caps) with spermicidal foam/gel/film/cream/vaginal suppository
• Total abstinence or (when this is in line with the preferred and usual lifestyle of the subject); periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception

You may not qualify if:

• If the patient is currently receiving erythroid stimulating agents (ESA) with plans to continue during study, less than 2 months duration of ESA prior to starting study drug and no dose escalation within 2 months of start of study drug
• If the patient is being treated with granulocyte-colony stimulating factor (GCSF) and/or a TPO-mimetic (for example, eltrombopag or romiplostim) with plans to continue during the study: Less than 2 months duration of GCSF or the TPO-mimetic treatment prior to starting study drug; or GCSF and/or TPO-mimetic has been added to ESA therapy within 2 months of start of study drug
• If patient is being treated with lenalidomide with plans to continue during the study: Stable dose for less than 3 months prior to start of study drug
• If patient is being treated with hypomethylating agents (HMA) (for example, azacitidine or decitabine) with plans to continue during the study: Stable dose for less than 6 months prior to start of study drug
• Currently enrolled in, or discontinued within the last 14 days from a clinical trial involving an investigational product or non-approved use of a drug, or concurrently enrolled in any other type of medical research judged not to be scientifically or medically compatible with this study
• Presence of \>= 10% blast by morphologic examination of bone marrow aspirate or biopsy
• Platelets =\< 50,000
• Microcytosis on screening blood cell count (CBC) (mean corpuscular volume \[MCV\] \< 81 fL)
• Active gastrointestinal (GI) ulceration or hemorrhage
• Have a serious preexisting medical condition that, in the opinion of the investigator would preclude participation in the study (for example a GI disorder causing clinically significant symptoms such as nausea, vomiting, and diarrhea, or malabsorption syndrome) or that would result in a life expectancy of less than 1 year
• Known hypersensitivity to deferasirox
• History of non-transfusional hemosiderosis
• Prior hematopoietic stem cell transplant for the diagnosis of MDS
• A second primary malignancy that in the judgment of the principal investigator (PI) or designee may affect the interpretation of results
• Have an active fungal, bacterial, and/or known viral infection including human immunodeficiency virus (HIV) or viral (A, B, or C) hepatitis

Sponsors & Collaborators

• Fred Hutchinson Cancer Center: lead
• National Cancer Institute (NCI): collaborator
• Novartis Pharmaceuticals: collaborator

Study Sites (1)

Fred Hutch/University of Washington Cancer Consortium

Seattle, Washington, 98109, United States

Location

MeSH Terms

Conditions

Anemia; Myelodysplastic Syndromes

Interventions

Deferasirox

Condition Hierarchy (Ancestors)

Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Benzoates; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Triazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Results Point of Contact

• Title: Dr. Bart Scott
• Organization: Fred Hutch Cancer Research Center

bscott@fredhutch.org

206.667.1990

Study Officials

• Bart Scott

  Fred Hutch/University of Washington Cancer Consortium

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 21, 2016
• First Posted: October 25, 2016
• Study Start: March 2, 2017
• Primary Completion: December 17, 2018
• Study Completion: December 17, 2018
• Last Updated: July 1, 2020
• Results First Posted: July 1, 2020

Record last verified: 2020-04

Locations

US (1)